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Published online 30 October 2007 | Nature | doi:10.1038/news.2007.204
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Gene therapy sees early success for neurodegenerative disease
Treatment uses HIV to insert genetic material into ALD patients.
Two children with a common neurodegenerative disease are seeing early signs of success from a pioneering gene-therapy treatment, researchers report this week.
The results raise hopes for a treatment for adrenoleukodystrophy (ALD), and, the researchers add, mark the first successful use of an attenuated HIV virus to carry a therapeutic gene into a patient’s cells.
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The European Leukodystrophy Association, known as ELA (http://www.ela-asso.com), largely contributed to fund Pr. Aubourg's research over the past 15 years. In addition to assistance and support for patients and families in Europe, ELA is the first private organization in the world to fund research on leukodystrophies (21 millions euros) with 3,7 millions dedicated to ALD research. Taken from the latest ELA press release: «"This first success brings a tremendous hope for the parents of ALD children, we are nevertheless aware that it's too early to proclaim victory and that this first test will have to be confirmed" indicates Guy Alba, the ELA founding president. Let's not forget that this trial can be carried out thanks to the progress made in AIDS research. The advancement on ALD brings now hopes for other diseases like leukaemia and Alzheimer disease. This proves that research on orphan diseases shouldn't be neglected. Because supposedly more complex, the investigators must show great imagination and audacity. They must innovate and it pays... » Marie-Josee Duran for the ELA Association